FAQ

Our frequently asked questions and answers are organized into groups:







Questions from donors and supporters



What do you call your cure?

Our patents fall under the title for the first one that we submitted: “Gene Regulation Therapy Involving Ferritin.”
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What are the costs associated with carrying our your plan to treating sickle-cell and related diseases?

The next cost hurdle is for further experiments to show that our cure is safe in animals and, in the same experiments, show that our ways of delivering the cure (as an injection or as a pill) will work in animals. This is also an expensive step on the way to clinical application. But it is an essential step before clinical trials in humans can be started.

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Is your cure patented?

Yes, patents have been granted to us and issued in the United States (7517669 and 7718699), and the European Union (25 EU countries): EP1354032B1, and Australia: AU2002217964.
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Why are patents necessary?

Patents are necessary to give a pharmaceutical company an exclusive license; otherwise, they will not invest in clinical trials and further developments necessary to bring a treatment to market and, therefore, to clinics to treat patients. Thus, we need to get over this “cost” hurdle to get our treatment to the bedside.

However, we are also considering partnerships with other globally active non-profits as a means of funding the world-wide distribution of our cure for SCD. In this case, patents may still be required in some countries, depending on how manufacuring and distribution of the medicine is handled.
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Does SCCF’s cure require gene therapy?

No. We call our approach to curing sickle cell gene regulation therapy, and the result is called a phenotypic cure. That is to say that no genes are permanently altered; instead, we use normal bodily signals to turn off the sickle cell and activate another gene with the same function in its place.

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How may I donate to the SCCF efforts?

Donations (as personal checks) may be sent to:
The Sickle Cell Cure Foundation, Inc.
755 Research Parkway, Suite 451 / Mailbox 24
Oklahoma City, OK 73103
Click here (or use the button at the upper right of SCCF’s home page) to make a donation using PayPal and a credit- or debit-card. More information may be found at this SCCF page or by telephoning (405) 922-5774.
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Is the SCCF recognized as a legitimate agency?

Yes, The Sickle Cell Cure Foundation, Inc. (SCCF) has a federal tax I.d. number, is registered in the State of Oklahoma as a Nonprofit Corporation, and has received 501(C(3) status from the IRS. Thus, all donations are tax-deductible. Donors receive a receipt that can be used for tax purposes.

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What is the process for developing a new drug?

  • Develop a conceptual model based on current research: a “thought experiment”.
  • Laboratory studies to verify the expected chemical and biological reactions
  • Fundraising and partnerships to accomplish the remaining steps.  Although grants and donations can accomplish the previous tasks, the remaining tasks are progressively more expensive to conduct.
  • Animal studies to test effectiveness and safety in living beings.  Sometimes referred to as a “Phase 0 clinical trial”, these investigations determine the effects of the drug in living bodies and the effects of the body on the drug (pharmacodynamics and pharmacokinetics).  The studies help identify appropriate measures of the drug’s effects, as well as how the living cells metabolize, alter, and eliminate the drug.  As of the start of 2014, this is where we were in the process.
  • Government Oversight — through the Investigational New Drug (IND) approval procedure designed by the US Food and Drug Administration (FDA).  As part of this process, a review board is created to oversee the testing and evaluation of the upcoming clinical trials.
  • Clinical trials
    • Phase 1: Screening for safety initial introduction of the drug into healthy human subjects where the product is tested for safety, dosage tolerance, absorption, metabolism distribution and excretion
    • Phase 2: Establishing the efficacy of the drug, usually against a placebo with a limited patient population in order to determine: (i) the efficacy of the product for specific, targeted indications; (ii) dosage tolerance and optimal dosage; and (iii) possible adverse side effects and safety risks
    • Phase 3: Final confirmation of safety and efficacy, further evaluate dose size and clinical efficacy, while further testing for safety in an expanded patient population at geographically dispersed clinical study sites
  • Regulatory approval

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Questions from sufferers and their families

The purpose of the Sickle Cell Cure Foundation is bring an inexpensive, effective, and safe treatment for sickle-cell anemia into widespread use. We are keenly aware of the suffering of victims and their families. As researchers, our ability to respond to individual needs for medical advice is limited. However, we offer the following questions and answers in an attempt to help people to find appropriate assistance while the search for more effective treatments continues.


How do I find a qualified sickle-cell physician?

Look for someone in your area with a specialty in “hematology,” the study of blood disorders. The American Society of Hematology provides an on-line database of registered hematologists: Find a Hematologist where you can search for specialists in your area. U.S.News & World Report has compiled a searchable list of doctors organized by specialty: Hematologists. The following list may lead families to experts in their country. 

Sickle Cell Foundation Nigeria
Bahrain Society for Sickle Cell Anaemia Patients Care
Sickle Cell Association of Uganda
The Sickle Cell Information Center provides an extensive list of world-wide contacts.
Sickle Cell Awareness Group of Ontario
Sickle Cell Foundation of Tanzania and the Muhimbili Wellcome Programme in Dar-es-salaam.
The US Center for Disease Control (CDC) assembled a state-by-state directory of sickle-cell organizations in 2011.



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What are the current treatments for sickle-cell disease and how do they differ?

The table below presents the commonly employed current treatments for comparison purposes and to help families to ask informed questions of their physicians.  This is not medical advice for particular patients or situations, but instead a description of the various treatment options.

Treatment Purpose  Other Effects  Cost and Duration
Antibiotics, such as penicillin Control secondary infections  Bowel disorders.  Risk of antibiotic resistent strains developing, if schedule not followed carefully.  Inexpensive, long term.
Painkillers, such as Paracetamol, acetaminophen, nonsteroidal anti-inflammatory drugs, and narcotics Alleviate pain. Risk of kidney damage from actaminophen.  Risk of narcotic addiction.  Varies.
Blood transfusion Prevent organ damage and alleviate pain  Long-term iron overload may occur. Crisis treatment.
Bone-marrow transplant  Cures sickle cell formation.  The procedure creates the risk of infection.  The transplanted tissue may be rejected.  Expensive, requires compatible donor who may be very difficult to find.
Diet modification, such as additional folic acid.  Reduce sickling of red blood cells. Seek advice from a physician.  Dangerous side effects sometimes occur.  Ongoing.
Chemotherapy, such as hydroxyurea  Lessen crisis frequency.  Potential increased anemia.  Potentially carcinogenic. Expensive daily dosage


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What can victims do about the pain?

First, sufferers need to avoid situations that have greater risk of triggering a painful episode.  These include:
Reduced body temperature, such as bathing in cold water or cold weather,
Dehydration, such as outdoor exertion, or
Lower ambient oxygen, such as higher altitudes, airplanes, or air pollution.

All these factors increase the risk of sickling or inhibit the production of red blood cells.  So, caution is advised in travel (especially by airplane), colder weather, exercise, and diet.  Avoiding too much salt, and drinking water copiously should help.

Once pain has begun, a few home-based tactics should help.  These suggestions are not intended to supplant expert medical care. Sickle-cell patients should obtain and follow the guidance of their local medical professionals.  Expert medical care always should be sought, because health-care providers have other options, such as oxygen, intravenous fluids, blood transfusion, hydroxyurea, and prescription pain medications.  Females should discuss hormone therapy with their caregiver, if painful menses occur.



Over-the-counter pain medication, such as ibuprofen, naproxen, or  acetaminophen.  Of course, dosage limitations (example) should be observed.
Heating pads or warm baths.
If air travel is necessary, investigate supplemental oxygen, and drink plenty of water.

 


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Why can’t we get the cure now?

The objective of our non-profit foundation is to demonstrate, prove, and distribute a safe, effective, and inexpensive treatment for sickle-cell disease. The staff are biologists and chemists with volunteer assistants, and our focus is to expedite the drug development. As yet, our selected approach is not yet proven safe and effective. Our team does not consist of practicing physicians and cannot treat and monitor patient health. Indeed, it would be unethical and illegal in the United States for us to do so.

However, we intend to conduct clinical trials. We cannot now predict when and where the first trials will occur. Before then, we must successfully complete ongoing animal trials, which will last at least another year or more (as of September, 2013).


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